Summary of Vutrisiran (Amvuttra) FDA Approval of amyloid cardiomyopathy (ATTR-CM).
Summary of Vutrisiran (Amvuttra) FDA Approval of amyloid cardiomyopathy (ATTR-CM).
1. Alnylam Pharmaceuticals received FDA approval for vutrisiran (Amvuttra) to treat transthyretin amyloid cardiomyopathy (ATTR-CM). It was previously approved for polyneuropathy of hereditary ATTR amyloidosis (hATTR-PN).
2. Clinical Trial Evidence: The approval was based on results from the HELIOS-B trial, published in The New England Journal of Medicine (August 2024). The study involved 655 patients, showing reductions in all-cause mortality, recurrent cardiovascular events, and improved quality of life.
3.Treatment Regimen & Cost: Vutrisiran requires four subcutaneous doses per year, with an annual cost of $477,000. The company expects insurance coverage for up to 99% of patients, similar to its coverage for hATTR-PN.
4. Growing ATTR-CM Treatment Market: Previously, Pfizer’s tafamidis (Vyndaqel, Vyndamax) was the only available ATTR-CM treatment. In November 2024, BridgeBio’s acoramidis (Attruby) was also approved, increasing treatment options.
6. Patient Advocacy Response: Amyloidosis Support Groups welcomed the approval, emphasizing the continued unmet needs of ATTR-CM patients and the positive impact of new treatment options.
